Welcome to Gene Therapy Net
Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials.
The Prickly Debate on Germline Gene Therapy, and Moving It Forward
Posted on: 26 June 2019, source: SingularityHub
In 2016, a healthy baby boy came screaming into the world in a Mexican clinic. Harboring DNA from three parents, the baby had had his genes dramatically altered while still an embryo. Without the treatment, an inheritable neurological disorder would’ve killed him before the age of three. Two years later across the world in China, two girls, also as embryos, had their genes edited using CRISPR to make them (theoretically) resistant to HIV infection. The edits didn’t hit their intended target—instead, the girls may be more prone to infections, have altered brain functions, and even experience earlier deaths.
In 2016, a healthy baby boy came screaming into the world in a Mexican clinic. Harboring DNA from three parents, the baby had had his genes dramatically altered while still an embryo. Without the treatment, an inheritable neurological disorder would’ve killed him before the age of three. Two years later across the world in China, two girls, also as embryos, had their genes edited using CRISPR to make them (theoretically) resistant to HIV infection. The edits didn’t hit their intended target—instead, the girls may be more prone to infections, have altered brain functions, and even experience earlier deaths.
Avexis Receives FDA Approval For Zolgensma, First And Only Gene Therapy For Pediatric Patients With Spinal Muscular Atrophy
Posted on: 25 May 2019, source: Novartis
AveXis, a Novartis company, today announced the US Food and Drug Administration (FDA) has approved Zolgensma® (onasemnogene abeparvovec-xioi) for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time intravenous (IV) infusion. Zolgensma is the first and only gene therapy approved by the FDA for the treatment of SMA, including those who are pre-symptomatic at diagnosis.
AveXis, a Novartis company, today announced the US Food and Drug Administration (FDA) has approved Zolgensma® (onasemnogene abeparvovec-xioi) for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time intravenous (IV) infusion. Zolgensma is the first and only gene therapy approved by the FDA for the treatment of SMA, including those who are pre-symptomatic at diagnosis.
NIH Streamlines Gene Therapy Oversight and Charts a Course for Considering Emerging Technology
Posted on: 26 April 2019, source: NIH
In August 2018, the National Institutes of Health and the Food and Drug Administration (FDA) announced a joint proposal to streamline duplicative and burdensome oversight over gene therapy. As part of this effort and following public input, NIH today released the amended NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules. The amendments remove the requirement to register and report on human gene therapy protocols under the NIH Guidelines to the NIH. Robust oversight continues under the FDA, which has regulatory oversight of all human gene therapy clinical trials. In addition, NIH-funded human gene therapy research remains subject to the usual NIH oversight that applies to all NIH-funded research, and oversight by local authorities such as Institutional Review Boards and Institutional Biosafety Committees.
In August 2018, the National Institutes of Health and the Food and Drug Administration (FDA) announced a joint proposal to streamline duplicative and burdensome oversight over gene therapy. As part of this effort and following public input, NIH today released the amended NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules. The amendments remove the requirement to register and report on human gene therapy protocols under the NIH Guidelines to the NIH. Robust oversight continues under the FDA, which has regulatory oversight of all human gene therapy clinical trials. In addition, NIH-funded human gene therapy research remains subject to the usual NIH oversight that applies to all NIH-funded research, and oversight by local authorities such as Institutional Review Boards and Institutional Biosafety Committees.
Novartis Says Baby's Death May Be Tied to Gene Therapy
Posted on: 24 April 2019, source: Bloomberg
Novartis AG said the death of a six-month-old baby in a study evaluating its experimental gene therapy Zolgensma was considered by the clinical trial investigator to be potentially related to the treatment. The death of the patient with a devastating disease known as spinal muscular atrophy occurred in a late-stage trial in Europe that’s still enrolling patients, the Swiss drugmaker said in an emailed statement. Initial findings show it involved a severe respiratory infection followed by neurological complications. The results of an autopsy are pending, and details have been reported to regulators, the company said.
Novartis AG said the death of a six-month-old baby in a study evaluating its experimental gene therapy Zolgensma was considered by the clinical trial investigator to be potentially related to the treatment. The death of the patient with a devastating disease known as spinal muscular atrophy occurred in a late-stage trial in Europe that’s still enrolling patients, the Swiss drugmaker said in an emailed statement. Initial findings show it involved a severe respiratory infection followed by neurological complications. The results of an autopsy are pending, and details have been reported to regulators, the company said.
First gene therapy to treat rare blood disease nears European approval
Posted on: 2 April 2019, source: STATnews
The first gene therapy to treat a rare blood disorder is one step closer to approval Friday following a recommendation by European officials. Lentiglobin, the gene therapy for beta-thalassemia developed by Cambridge, Mass.-based Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use (CHMP), the drug-reviewing arm of the European Medicines Agency. A final approval decision is expected within the next three months.
The first gene therapy to treat a rare blood disorder is one step closer to approval Friday following a recommendation by European officials. Lentiglobin, the gene therapy for beta-thalassemia developed by Cambridge, Mass.-based Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use (CHMP), the drug-reviewing arm of the European Medicines Agency. A final approval decision is expected within the next three months.
Should gene therapy patients be asked to pay?
Posted on: 28 March 2019, source: evaluate.com
The price in value-based payment deals should by definition be justified, so why is patient cost-sharing part of the calculation? Spark covers this cost, but there is no guarantee that all gene therapy companies will. As Novartis’s gene therapy Zolgensma nears a US FDA decision, the sector has only a vague idea of how much the Swiss group will charge for the spinal muscular atrophy (SMA) treatment, other than it will be a number followed by six zeroes. Just as importantly, the families with children suffering from this fatal condition do not know what, if anything, they will be asked to pay. However, there are some clues from the way in which the first US-approved gene therapy, Spark Therapeutics’ Luxturna, was financed.
The price in value-based payment deals should by definition be justified, so why is patient cost-sharing part of the calculation? Spark covers this cost, but there is no guarantee that all gene therapy companies will. As Novartis’s gene therapy Zolgensma nears a US FDA decision, the sector has only a vague idea of how much the Swiss group will charge for the spinal muscular atrophy (SMA) treatment, other than it will be a number followed by six zeroes. Just as importantly, the families with children suffering from this fatal condition do not know what, if anything, they will be asked to pay. However, there are some clues from the way in which the first US-approved gene therapy, Spark Therapeutics’ Luxturna, was financed.
New Gene Therapy Shows Promise For Patients With Sickle Cell Disease
Posted on: 9 March 2019, source: CBS News
An experimental and innovative type of gene therapy is being used to attempt to cure sickle cell anemia. The director of the National Institutes of Health says the results of an NIH clinical trial on a gene therapy for sickle cell anemia "looks like a cure" for the painful and often deadly genetic disease that affects 100,000 Americans, with nearly all being African Americans. 60 Minutes follows the trial for more than a year, focusing on a Florida woman who now appears to be rid of sickle cell anemia. Dr. Jon LaPook's report will be broadcast on 60 Minutes, Sunday, March 10 at 7:00 p.m. ET/PT on CBS.
An experimental and innovative type of gene therapy is being used to attempt to cure sickle cell anemia. The director of the National Institutes of Health says the results of an NIH clinical trial on a gene therapy for sickle cell anemia "looks like a cure" for the painful and often deadly genetic disease that affects 100,000 Americans, with nearly all being African Americans. 60 Minutes follows the trial for more than a year, focusing on a Florida woman who now appears to be rid of sickle cell anemia. Dr. Jon LaPook's report will be broadcast on 60 Minutes, Sunday, March 10 at 7:00 p.m. ET/PT on CBS.
Gene Therapy News
- The Multibillion-Dollar Race for Gene Therapy Manufacturing Is On - Yahoo Finance
Wed, 17 Jul 2019 19:22 GMT - Details emerge of first Huntington's disease gene therapy clinical trial - HDBuzz
Wed, 17 Jul 2019 14:30 GMT - Gene therapies: Up and coming ... and expensive - Healio
Wed, 17 Jul 2019 13:14 GMT - Reversing the risk of sudden cardiac death with gene therapy - FierceBiotech
Wed, 17 Jul 2019 12:15 GMT - New insight into microRNA function can give gene therapy a boost - Science Daily
Wed, 17 Jul 2019 16:56 GMT
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Recent early view article in Journal of Gene Medicine:
You can buy a unique research paper online on gene therapy topics here.